CN106488984B - 一种治疗周围神经病变以及运动神经元疾病的方法 - Google Patents

一种治疗周围神经病变以及运动神经元疾病的方法 Download PDF

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CN106488984B
CN106488984B CN201580020318.9A CN201580020318A CN106488984B CN 106488984 B CN106488984 B CN 106488984B CN 201580020318 A CN201580020318 A CN 201580020318A CN 106488984 B CN106488984 B CN 106488984B
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aav
composition
vector
nucleic acid
muscular atrophy
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CN106488984A (zh
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A·M·布伊贝洛
M·K·奇尔德斯
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Genethon
Wake Forest University Health Sciences
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Wake Forest University Health Sciences
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201580020318.9A 2014-04-18 2015-04-20 一种治疗周围神经病变以及运动神经元疾病的方法 Expired - Fee Related CN106488984B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14165329.5A EP2933335A1 (en) 2014-04-18 2014-04-18 A method of treating peripheral neuropathies and motor neuron diseases
EP14165329.5 2014-04-18
PCT/EP2015/058505 WO2015158924A1 (en) 2014-04-18 2015-04-20 A method of treating peripheral neuropathies and motor neuron diseases

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CN106488984A CN106488984A (zh) 2017-03-08
CN106488984B true CN106488984B (zh) 2020-01-07

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US (1) US20170071972A1 (ja)
EP (2) EP2933335A1 (ja)
JP (1) JP2017513861A (ja)
CN (1) CN106488984B (ja)
CA (1) CA2945558A1 (ja)
WO (1) WO2015158924A1 (ja)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2016073693A2 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3458589A4 (en) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
US20200383590A1 (en) * 2016-11-17 2020-12-10 Medici Technologies Llc Methods and Apparatuses for Central Venous Pressure Measurement Status
RU2665771C1 (ru) * 2017-03-17 2018-09-04 Общество с ограниченной ответственностью "Медсервис" Средство для лечения состояний человеческого организма, связанных с уменьшением уровня экспрессии гена ang и/или уменьшением количества и/или активности белка ангиогенина на основе генно-терапевтических субстанций с геном ang, способ получения и использования
WO2018189208A1 (en) * 2017-04-10 2018-10-18 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP2020532284A (ja) * 2017-07-08 2020-11-12 ジェネトン 脊髄性筋萎縮症の治療
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
CA3086942A1 (en) 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements
JP7478675B2 (ja) * 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
WO2020091235A1 (ko) * 2018-10-30 2020-05-07 사회복지법인 삼성생명공익재단 Fbln5 유전자 결손 동물 모델 및 이를 이용한 신경 또는 근육 질환 치료제 스크리닝 방법
KR102360896B1 (ko) * 2018-10-30 2022-02-09 사회복지법인 삼성생명공익재단 Fbln5 유전자 결손 동물 모델 및 이를 이용한 신경 또는 근육 질환 치료제 스크리닝 방법
WO2020208032A1 (en) 2019-04-08 2020-10-15 Genethon Hybrid promoters for muscle expression
CN110616192B (zh) * 2019-08-09 2021-11-09 无锡傲锐东源生物科技有限公司 一种抗人神经丝轻链(nefl)的单克隆抗体及其应用
CN110511992B (zh) * 2019-08-27 2023-06-06 深圳市宝安区妇幼保健院 一种tardbp突变基因、检测引物和试剂盒
CN112852880B (zh) * 2021-01-28 2022-12-06 中吉智药(南京)生物技术有限公司 一种基于诱导型昆虫细胞生产aav基因药物的方法
CN117897492A (zh) 2021-08-04 2024-04-16 吉尼松公司 用于肌肉和cns中的基因表达的杂合启动子

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1950397A (zh) * 2004-03-30 2007-04-18 Ns基因公司 生长因子NsG33的治疗用途
CN101883859A (zh) * 2007-10-05 2010-11-10 吉尼松公司 使用外周注射aav载体向运动神经元进行广泛基因投送
WO2010129021A1 (en) * 2009-05-02 2010-11-11 Genzyme Corporation Gene therapy for neurodegenerative disorders

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20010009904A1 (en) * 1997-12-30 2001-07-26 Jon A. Wolff Process of delivering a polynucleotide to a cell via the vascular system
US5780489A (en) * 1996-08-21 1998-07-14 Brooks; Benjamin Rix Method for treating amyotrophic lateral sclerosis
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
DK1453547T3 (en) 2001-12-17 2016-12-05 Univ Pennsylvania ADENOASSOCATED VIRUS (AAV) SEROTYPE 8 SEQUENCES, VECTORS CONTAINING THESE AND APPLICATIONS THEREOF
AU2004278684B2 (en) 2003-09-30 2011-05-12 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
EP1691609A4 (en) * 2003-12-11 2006-11-29 Mirus Bio Corp RELEASE OF VIRUS VECTORS ANEXTRAVASAL PARENCHYM CELLS
US20050129660A1 (en) * 2003-12-11 2005-06-16 Hagstrom James E. Process of delivering a virally encapsulated polynucleotide or viral vector to a parenchymal cell via the vascular system

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1950397A (zh) * 2004-03-30 2007-04-18 Ns基因公司 生长因子NsG33的治疗用途
CN101883859A (zh) * 2007-10-05 2010-11-10 吉尼松公司 使用外周注射aav载体向运动神经元进行广泛基因投送
WO2010129021A1 (en) * 2009-05-02 2010-11-11 Genzyme Corporation Gene therapy for neurodegenerative disorders
CN102459611A (zh) * 2009-05-02 2012-05-16 建新公司 神经退行性疾病的基因治疗

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
"Hydrodynamic Limb Vein Injection of Adeno-Associated Virus Serotype 8 Vector Carrying Canine Myostatin Propeptide Gene into Normal Dogs Enhances Muscle Growth";Chunping Qiao等;《HUMAN GENE THERAPY》;20090114;第20卷(第1期);具体参见对比文件1摘要，第2页右栏第3段，第3页右栏倒数第2段 *
"Intrajugular vein delivery of AAV9 –RNAi prevents neuropathological changes and weight loss in Huntington’s disease mice";Brett Dufour等;《Molecular therapy》;20140106;第22卷(第4期);第797-810页 *
"Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice";DM McCarty等;《Gene Therapy》;20090709;第16卷(第11期);第1340-1352页 *
"Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates";Steven J Gray等;《Molecular Therapy》;20110411;第19卷(第6期);第1058-1069页 *

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Publication number Publication date
CN106488984A (zh) 2017-03-08
WO2015158924A1 (en) 2015-10-22
JP2017513861A (ja) 2017-06-01
EP2933335A1 (en) 2015-10-21
CA2945558A1 (en) 2015-10-22
EP3132042B1 (en) 2019-02-20
EP3132042A1 (en) 2017-02-22
US20170071972A1 (en) 2017-03-16

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